

Unlocking Transcription Factors to Address Unmet Medical Need
Developing small molecules with potential across multiple serious diseases

Advancing Oral Transcription Factor Therapies for Patients with Limited Options
At Altay Therapeutics, we're unlocking the therapeutic potential of transcription factor inhibition to address serious, underserved diseases. Our pipeline includes the first oral small molecule inhibitor of DUX4 for facioscapulohumeral muscular dystrophy (FSHD) and a potential best-in-class STAT3 inhibitor with broad potential in cancer and chronic inflammatory diseases. We aim to bring novel transcription factor inhibitors to the clinic, not just to advance the science, but to offer differentiated therapeutic options to patients.

Unlocking theTherapeutic Power of Transcription Factors
Transcription factors regulate the expression of hundreds of genes and play central roles in development, immunity and disease. Yet they've long been considered 'undruggable' due to their lack of defined binding pockets and complex structural dynamics.
At Altay, we've developed a therapeutic area-agnostic drug discovery platform that challenges this notion. By identifying novel allosteric sites - emerging only through dynamic structural changes in transcription factors - we design highly-selective small molecule inhibitors with the potential to be first- or best-in-class.
A Pipeline Rooted in Precision Biology
Inhibitor
program
indication
STAT3
Inhibitor
Inflammatory
diseases
& CANCER
Research &
discovery
Programs
oncology
Validation
Optimization
CANDIDATE
ENABLING
Phase 1/2a
2026
DX5057 DUX4 Inhibitor
FSHD
IND
ENABLING
PHASE 1/2A
2025
2026
STAT3 Inhibitor
Inflammatory
diseases & oncology
DEVELOPMENT
CANDIDATE
IND ENABLING
2026
Research & discovery Programs
ONCOLOGY
Lead Optimization
DX5057 for FSHD
Our lead program targets DUX4, the transcription factor that drives FSHD pathology. DX5057 is the first oral small molecule inhibitor of DUX4, showing preclinical efficacy comparable to the leading IV administered gene-targeting therapies, with the added benefit of ease of use and scalable delivery.
STAT3 Inhibitor for Inflammatory Diseases and Oncology
Undisclosed Program in Oncology
We are supported by a team of esteemed advisors and collaborators who bring a wealth of drug development expertise to Altay
Press Releases, Media & Presentations
JUN 2025
Altay is selected as a Finalist for the $10M FSHD Bonus Prize alongside the $101M XPrize Healthspan competition
We're honored to have been selected as a Finalist for the $10M FSHD Bonus Prize alongside the $101M XPrize Healthspan competition. Read about it here.
Dec 2023
Altay is awarded a Golden Ticket award by Astellas Therapeutics!
We're honored to have been selected as a Golden Ticket recipient by Astellas Therapeutics! Read about it here.
Sep 2022
SOLVE FSHD Invests in Altay Therapeutics to develop novel inhibitors for FSHD
SOLVE FSHD's investment will accelerate our drug development efforts and shorten the time to enter clinical trials. Read about it here.
Nov 2021
Altay is awarded a Golden Ticket by Bristol Myers Squibb!
We're honored to have been selected as a Golden Ticket recipient by Bristol Myers Squibb! Read about it here.

We'd love to hear from you.
Feel free to reach out to us with any questions or inquiries.